May 07, 2010

The Importance of Clinical Trials

A recent supplement in the Wall Street Journal discussed the importance of clinical trials, and provided an introduction to the clinical research process. The guide was written by various researchers and physicians in the clinical research community, and included segments from groups such as the Association of Clinical Research Professionals (ACRP).

Clinical Trials

Most commonly, clinical trials are used to test the safety and effectiveness of drugs and devices. Usually, they are sponsored by pharmaceutical companies and are conducted by research teams that include doctors and other medical professionals. Typically, trials are typically conducted in four phases:

-Phase one is when generally healthy people are given the medication to test if the ingestion of the pill or treatment will have no adverse, toxicological effect;

-Phases two and three dive deeper into the safety, effectiveness, and dosage of the medication, and it’s after these stages when the FDA would approve the drug or device; and

-Phase four examines new uses for previously approved treatments.

All of the phases in clinical trials are governed by strict protocols, and are overseen by many regulatory bodies, from the Food and Drug Administration (FDA) to small Independent Review Boards (IRBs). IRBs are a group of independent medical experts, ethicists, as well as lay people. Researchers report periodically to the IRB, outlining such things as contact with patients, the tests conducted, the results recorded and even the side effects reported. IRBs are accredited by the Association for the Accreditation of Human Research Protection Programs (AAHRPP).

As Ken Getz, founder and chairman of the Centerfor Information and Study on ClinicalResearch Participation (CISCRP) noted, “there’s no question that clinical trials will play a large and growing role in the medical options” patients have today and in the future. In fact, nearly 4,000 experimental drug therapies are in active clinical trials today and that number will continue to grow as improvements are made in detecting disease, in understanding the root causes of acute and chronic illnesses, and in discovering medical innovations.

Mr. Getz also predicted that “in the not-so-distant future it will be more common for clinical trials to be discussed during routine visits with the doctor as electronic health records and clinical research converge.” This prediction is much different from how most people stumble upon clinical trials today.

Usually, patients learn about clinical trials only “when faced with the sudden prospect of a serious, often life threatening, illness for which no marketed medication is available or adequate.” Since patients choose to wait, they are forced to rush through information about clinical trials they have received from a physician or nurse or personal search on the Internet, which can leave them feeling overwhelmed and confused. If patients were able to take a closer look at the impact clinical trials have on the lives of volunteers and future generations, such decisions would not be as hard.

Impact of Clinical Trials

It is no coincidence today that “the age-adjusted death rate in the US for coronary heart disease was cut in half from 1980-2000” because much of this progress was the result of investing in basic and clinical research. In fact, while half of the decrease is attributed to reductions in cholesterol levels, blood pressure, and smoking, the other “half of this decrease can be directly attributed to medical therapies validated in clinical trials.”

The overwhelming majority of these trials were made possible because of the “resources spent on clinical development of new therapies, which have been provided by pharmaceutical, biotechnology, and device companies.” As Judith M. Kramer, MD, MS, an associate professor of medicine at Duke University noted, “without the contributions of these organizations, along with those of the health professionals and patients who participate in clinical trials, public health in the US would not be what it is today.”

Critics of clinical trials believe they are unnecessary, profit-driven “experiments” conducted on humans. What these people do not realize is that “doctors don’t always know what treatment is best because objective comparison of large numbers of patients is needed to sort out the truth about benefits and risks.” As a result, doctors recommend patients volunteer in clinical trials only when it is unclear which treatment option being tested is best. But patients should not be reluctant to volunteer for clinical trials because as history shows, they have saved the lives of millions.

For example, it was a randomized trial of the Salk polio vaccine in over 600,000 school children that led to the approval of the first preventive treatment for that disease, and with the later addition of an oral vaccine, polio has been nearly eradicated in the US. Likewise, measles was nearly eliminated by a vaccine tested in clinical trials, and clinical trials and their participants also contributed to the scientific foundation for tuberculosis policies still adhered to today. Consequently, without clinical trials “these diseases would still be a danger to America’s children today.”

Since the success from clinical trials has led to numerous benefits for patients, it was noted that more funding is needed for cancer research. Without the necessary funds, the pace of research will slow; key discoveries will be delayed; and the implementation of new strategies to control cancer will take years longer than necessary.

Finding money during the economic downturn has played a role in reduced research funding. Some people believe that industry is conducting clinical research in developing countries to make up for decreases in funding, and to increase profits. “This is rarely the case because researchers improve the standard of care in these areas by giving medical training, leaving behind valuable equipment, and forming partnerships with communities.”

Additionally, companies are not given many other options to find ways to keep funding research, especially when NIH changes its process for funding research by requiring groups to compete for taxpayer dollars devoted to clinical trials whereas before they were given the money as grants. As a result, industry will have to add even more than the 90 percent of funding for research it already provides.

The reality is, industry takes trials off shore because they are able to do more, find new cures and better treatments with less. And companies are happy to do this for patients because a lot of pressure is “being placed on research professionals to be sure they’re doing their work as efficiently as possible.”

Patients

The supplement also included comments from patients who have participated in clinical trials. One patient noted that “taking part in trials made her feel like she had some control over the course of her disease.” When faced with the tragic news of illness, patients overwhelmingly would take the chance of doing something rather than nothing. As one patient noted, she ““felt very threatened by this disease and wanted to take aggressive steps to fight it.”

That is why clinical trial participants told others to “have an open mind to being a clinical trial subject because you can learn all about the trial’s purpose and requirements and then decide whether to go for it.” Whether taking medicine or participating in a clinical trial, the reality is “people are just as likely to experience side effects while taking approved medication as they would be taking trial compounds.”

But participants can experience several benefits when involved in clinical trials, such as new treatment options, which are important because most participants are not satisfied with their current treatment. And if “a risk is known, that risk has to be reported to the ethics board and to the investigators conducting the study so people are apprised of what information has surfaced about that product.”

Conclusion

Behind every medicine and intervention that people have ever taken, are thousands of patients who have volunteered to participate in clinical trials, which have led to many breakthroughs in disease prevention and treatment in the last half-century. Without the willingness of these individuals, many would have suffered.

It is also important to recognize that “clinical research is not always devoted to finding the next “blockbuster” drug. Clinical trials also can contribute invaluable information about the benefits and safety of existing therapies, providing doctors and patients with reliable information for choosing between alternative treatments.

Ultimately, because “every medicine or medical device must be fully vetted through closely monitored and highly regulated clinical trials to insure their safety and effectiveness,” patients receiving medical care should be encouraged to participate in clinical trials.

For those people who participate in clinical trials, they are the heroes that are helping to develop the new drugs, devices, biologics, and treatments for the future, and improving the care of all Americans.